Parkinson’s ResearchersAttend a Gaucher MeetingWHY?

I work on Parkinson’s disease which affects half a million Americans… including, Michael J Fox, Mohamed Ali, Janet Reno as well as Pope John Paul II. Gaucher’s disease, in contrast, affects between 5,000 and 10,000 Americans.

The answer is simple: 5 years ago, scientists, led by Ellen Sidransky at the National Institutes of Health, found that some cases of Parkinson’s disease were predisposed to by the same defective gene, glucosecerebrosidase, that causes Gaucher’s. This tells us all that something about the causation of the two diseases is similar. So the answer is… I came to learn!! Gaucher’s researchers have been studying this gene for years and have learnt a lot about its function in cells, and how loss of that function damages cells and can kill them. I want to understand what they already know. I also came to teach Gaucher’s researchers what I know about Parkinson’s. We Parkinson’s researchers can learn a lot from the enormous amount of knowledge Gaucher’s researchers have understood about their disease and we can also explain what we already know about Parkinson’s disease and see how much of that knowledge might be useful for Gaucher’s disease.

Perhaps most useful, we can also help bring much needed money into Gaucher’s research. Before scientists realised the two diseases were related the amount of research effort into Gaucher’s was very small because it affected so few people… now, the amount of research into Gaucher’s is becoming more well-funded… and, this is desperately needed.

Scientists studying the brain. One group from the Parkinson's field, one group from the Gaucher field. The connection between the two diseases is being pursued.

There is so much we still do not know about both diseases. In particular, we really don’t understand why in some people Gaucher’s disease is so mild and in others so severe even if they have the same genetic defect. For us to understand this we need to study the disease in hundreds of Gaucher’s patients and this will cost a lot of money… perhaps the fact that this knowledge will also benefit the hundreds of thousands of people with Parkinson’s disease will help us get the funding we need to do this.

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